Individualized, Target-Driven Treatment Of Children With Idiopathic Short Stature

This study has been completed.

Sponsor:

Pfizer

ClinicalTrials.gov Identifier:

NCT00396097

First Posted: November 6, 2006

Last Update Posted: April 5, 2016

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To demonstrate that an individualized, formula-based Genotropin regimen for children with Idiopathic Short Stature will lead to a targeted height gain (to reach the target of 10th percentile (%), or -1.3 SDS) during 24 months of treatment. The endpoint at 4 years is to explore treatment efficiency over four years of two formula-based dose regimens (sub-arms) compared to standard treatment

A Four Year Open Label Multi Center Randomized Two Arm Study Of Genotropin In Idiopathic Short Stature Patients: Comparing An Individualized, Target Driven Treatment Regimen To Standard Dosing Of Genotropin

This was defined as an absolute difference between the 24-month height standard deviation score (SDS) and targeted 24-month height SDS (10th percentile (%), or -1.3 SDS). SDS indicates how similar the participant was to the reference population. These were calculated using 2000 Center for the Disease Control (CDC) growth reference tables (by age and gender).

Secondary Outcome Measures:

Variability of Height SDS at 24 Months [ Time Frame: 2 years ]

The continuous endpoint of variability of height SDS at 24 months was defined as the SD of the 24 month height SDS.

Time Cost (Months Until >= -2 SDS) [ Time Frame: 2 years ]

Time cost was defined as the number of months needed until height SDS was within the normal limit (ie, >= -2SDS).

Computed Cost of Height Gain at 48 Months [ Time Frame: 4 years ]

The computed cost of height gain was defined as the amount of drug used relative to the observed height-gain, in terms of mg/cm, this was calculated at Month 48.

Estimated Cost of Height Gain Estimated Until Full Adult Height (FAH) at 48 Months [ Time Frame: 4 years ]

The estimated cost of long-term height gain until FAH was calculated.

Change From Baseline in Height SDS at 48 Months. [ Time Frame: 4 years ]

Compare daily injections of formula-based HGH treatment to daily injections of standard HGH treatment in subjects with Idiopathic Short Stature over 24 months period followed by an exploratory 24 months period.

Active Comparator: Formula-based

Formula-based dose regimen

Drug: Genotropin

Compare daily injections of formula-based HGH treatment to daily injections of standard HGH treatment in subjects with Idiopathic Short Stature over 24 months period followed by an exploratory 24 months period.

Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:

3 Years to 10 Years (Child)

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

No

Criteria

Inclusion Criteria:

Prepubertal children with bone ages between 3 and 10 years of age for males and 3 and 9 years of age for females

Naive to Growth Hormone treatment

Exclusion Criteria:

Abnormal karyotype. Small Gestational Age and Skeletal dysplasia.

Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00396097